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Lufepirsen

Phase 2

Persistent Corneal Epithelial Defect | Small molecule | Ophthalmology |Glaukos Corporation|Last Updated: Jul 24, 2025

Target and mechanism

Molecular targetGJA1
Target class-Sen (Aso)
ModalitySmall molecule

Also known as lufepirsen high dose

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment84

FDA Designations

No designations recorded

Clinical trial landscape

Lufepirsen · 1 trial · 1 indication

Phase 2 1
NCT05966493A Study to Evaluate the Safety and Efficacy of NEXAGON® (Lufepirsen Ophthalmic Gel) in Subjects With PCEDPersistent Corneal Epithelial Defect
RECRUITING84 Analytics
PHASE2RECRUITING
A Study to Evaluate the Safety and Efficacy of NEXAGON® (Lufepirsen Ophthalmic Gel) in Subjects With PCED
Persistent Corneal Epithelial DefectUnlock trial analytics

Study Endpoints

Primary Endpoints

Achieve Corneal Re-epithelialization Including Durability (CRC)
End of Study: 28 Days after achieving re-epithelialization

The proportion of subjects achieving corneal re-epithelialization that is maintained for a minimum of 28 days, based on assessment of corneal fluorescein staining images of the PCED by a Central Reading Center (CRC).

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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
NEXAGON® (lufepirsen ophthalmic gel) High Dose ConcentrationEXPERIMENTALLufepirsen (High dose concentration) applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks.
NEXAGON Vehicle (ophthalmic gel)PLACEBO_COMPARATORVehicle applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks.
NEXAGON® (lufepirsen ophthalmic gel) Low Dose Concentration (EU sites only)EXPERIMENTALLufepirsen (Low dose concentration) applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks. (EU sites only).

Interventions

NameTypeDescription
lufepirsen high doseDRUGLufepirsen is an unmodified connexin43 antisense oligonucleotide.
VehicleDRUGMatching vehicle without lufepirsen.
lufepirsen low doseDRUGLufepirsen is an unmodified connexin43 antisense oligonucleotide.
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Eligibility Criteria

Age Range2 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites28

Inclusion Criteria: 1. Have a PCED that is at least 2 weeks in duration and refractory to one or more conventional non-surgical standard of care (SOC) treatments 2. Have no clinical evidence of improvement in the PCED within 2 weeks prior to randomization despite the use of non-surgical SOC treatme...

Countries:United StatesGermanyItalySpain
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Frequently asked questions about Lufepirsen

What is lufepirsen high dose used for?

Lufepirsen high dose is an investigational small molecule being developed for Persistent Corneal Epithelial Defect (PCED), a condition in ophthalmology. It is formulated as an ophthalmic gel and is currently in Phase 2 clinical development. The drug is not yet approved and remains under investigation for this indication.

What does lufepirsen high dose target?

Lufepirsen high dose targets GJA1, a gene that encodes connexin 43, a gap junction protein. As an antisense oligonucleotide (ASO), it is designed to modulate GJA1 expression. This molecular target is being studied for its potential role in treating Persistent Corneal Epithelial Defect.

Who makes lufepirsen high dose?

Lufepirsen high dose is being developed by Glaukos Corporation, a company traded on the NYSE under the ticker GKOS. Glaukos is conducting clinical research on this ophthalmic gel for Persistent Corneal Epithelial Defect.

What phase is lufepirsen high dose in?

Lufepirsen high dose is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The ongoing Phase 2 trial is actively recruiting participants to evaluate the safety and efficacy of the treatment for Persistent Corneal Epithelial Defect.

What clinical trials is lufepirsen high dose in?

Lufepirsen high dose is being studied in one active Phase 2 clinical trial with the identifier NCT05966493. This randomized, double-blind, placebo-controlled study is evaluating the safety and efficacy of NEXAGON (lufepirsen ophthalmic gel) in subjects with Persistent Corneal Epithelial Defect. The trial plans to enroll 84 participants across the United States, Germany, Italy, and Spain.

Is lufepirsen high dose the same as NEXAGON?

Yes, lufepirsen high dose is the same as NEXAGON. In clinical trials, the drug is referred to as NEXAGON (lufepirsen ophthalmic gel). Both names refer to the same investigational treatment being developed for Persistent Corneal Epithelial Defect.